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Rapid Expert Consultation on Critical Federal Capabilities Needed to Evaluate Real-World Safety, Effectiveness, and Equitable Distribution and Use of Medical Countermeasures During a Public Health Emergency
Rapid Expert Consultation on Critical Federal Capabilities Needed to Evaluate Real-World Safety, Effectiveness, and Equitable Distribution and Use of Medical Countermeasures During a Public Health Emergency
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Hodge, Jr.6 Ali S Khan7 Nicolette Louissaint8 Nicole Lurie9 Jewel Mullen10 Jennifer Nuzzo11 Tener Veenema12 This rapid expert consultation was produced through the Standing Committee for CDC Center for Preparedness and Response (SCPR)
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.13 MCMs deployed under regulatory access mechanisms, like EUAs in PHEs, must generally show some evidence of safety and efficacy under pre-market, well-controlled clinical trials (i.e., randomized controlled trials for medical products) , where available.
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Specifically, the consultation will confirm or modify the necessary federal capabilities to assess and monitor MCMs that are made available under appropriate regulatory access mechanisms, such as Emergency Use Authorizations, and are performing under real world conditions during a public health emergency. This effort will build upon the 2017 National Academies workshop Building a National Capability to Monitor and Assess Medical Countermeasure Use During a Public Health Emergency, and will draw from expert input, published literature, and lessons learned from the ongoing COVID-19 pandemic.
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BOX 1 Summary of National Academies Proceedings Focused on Monitoring and Assessing Medical Countermeasures and Real-World Evidence Building a National Capability to Monitor and Assess Medical Countermeasure Use During a Public Health Emergency: Going Beyond the Last Mile: Proceedings of a Workshop (2017) As part of the United States' scientific and research preparedness enterprise, there is an imperative to go "beyond the last mile" of MCM dispensing and administration to build and maintain a national capability to monitor and assess the use of MCMs (e.g., safety, compliance, clinical benefit)
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This workshop, the third of a three-part series titled Examining the Impact of Real-World Evidence on Medical Product Development, examined and suggested approaches for operationalizing the collection and use of RWE. It was designed to examine the current system of evidence generation and its limitations, to identify when and why RWE may be an appropriate type of evidence on which to base decisions, to learn from successful initiatives that have incorporated RWE, and to describe barriers that prevent RWE from being used to its full potential.
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. BOX 2 Previous National Academies Work Addressing Considerations for Data Systems and Data Used for Monitoring and Assessing Medical Countermeasure Use and Performance Comprehensive, Unified Data Management System from Rapid Expert Consultation on Data Elements and Systems Design for Modeling and Decision Making for the COVID-19 Pandemic These points serve as the basis for the necessary data elements, sources of data, gaps in collection, and suggestions for data system design and integration to improve modeling and decision making for COVID-19.
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3–4. Evaluation of Initial Draft Framework A draft list of key federal capabilities to monitor and assess MCMs was provided to the authors of this rapid expert consultation for their review (CDC, 2022b)
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Understanding current efforts under way or the current state – Establishing a comprehensive understanding of the current state of federal capabilities for evaluating RWE of MCMs that have been developed and were successful, that have been developed and did not work, are in the process of being developed, and those yet to be developed would assist in articulating a future state in context of the current state of federal capabilities. Achieving a balanced focus on each MCM type – While capabilities may apply broadly to multiple types of MCMs, sufficient detail of specific considerations tailored to each type of MCM is needed to facilitate a complete understanding through monitoring and assessment, due to differing standards for evidence generation depending on the specific MCM.
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21. In taking a CONOPS approach, the authors offer an organizing structure of an overarching goal, capabilities to achieve that goal, and core considerations as part of the framework of federal capabilities needed to evaluate real-world safety, effectiveness, and equitable distribution and use of MCM during a PHE (see Figure 1)
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An example of a goal statement for the purpose of monitoring and assessing MCMs could be: The goal of federal capabilities to evaluate RWE of MCMs in a PHE is to monitor and assess their safety, effectiveness, and equitable distribution and use, so that policy makers can make more rapid and effective decisions, communicate more accurate information to the public, and build or sustain community relationships and trust. This goal statement encompasses MCM distribution and use, because equity and other monitoring and assessment parameters are dependent on knowing where the MCM is in the system, and who specifically gets and ultimately uses the MCM.
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Evidence generation comprises: o Data Collection – The ability to collect, collate, and share valid and reliable information from multiple data sources. o Data Analysis – The ability to synthesize and analyze information in real time to inform decision making as a threat evolves.
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, and health information exchange standards for interoperability, the system can integrate and exchange the multiple sources and types of data (e.g., genomic; consumer- and patient-level quantitative and qualitative data; surveillance- and population-level data; place-based geocoded data; program data, such as manufacture, supply chain, allocation data, claims data, death records, survey data, sociodemographic data, wastewater surveillance, and qualitative community assessments) between different federal agencies as well as between federal agencies and STLT, community, and industry counterparts.
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Additionally, the authors determined that some of the initial, draft capabilities were relevant to product development not monitoring and assessment (e.g., develop partnerships with diagnostic developers to support rapid development, authorization of diagnostics for clinical use during PHEs, pre-identified lead for initial diagnostic reagent production, ability to scale production up or down included in federal contracts, and clarity on the use of the Stafford Act for development and validation of diagnostics) and should be classified as such.
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Systems to monitor claims data, Systems to implement relevant elements Engagement channels with groups who Systems to assess the legal options EHRs, prescription data, social media, of culturally and linguistically and ability routinely capture community-level data consistent with First Amendment limitations on qualitative, citizen science data, and appropriate standards to increase (e.g., universities, community-based free speech as well as other laws and policies federal MCM distribution. communication equity.
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provide sustained funding for community-level qualitative research efforts. NOTE: CBO = community-based organization; CEAL = Community Engagement Alliance16; DMI = Data Modernization Initiative17; EHR = electronic health record; EUA = Emergency Use Authorization; MCM = medical countermeasure; MOU = memorandum of understanding; NGO = nongovernmental organization; NIH = National Institutes of Health; RADx-UP = Rapid Acceleration of Diagnostics for Underserved Populations; RWD = real-world data; RWE = real-world evidence; STLT = state, tribal, local, or territorial public health agency: USCDI = United States Core Data for Interoperability.
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Public health, laboratory, and clinical trial networks with clinical study capacity to support federal comparative evaluation in real-world settings during PHEs. o Pre-determined minimum data elements, sets, and variables with consensus definitions determined with key stakeholders that will be reported on MCM safety, effectiveness, and equitable use.
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, and primary care research and community health networks to leverage their data and tools. Systems Systems to leverage, coordinate, and integrate federal data-collection systems and programs that were developed prior to and during the COVID-19 pandemic to monitor and assess MCM safety, effectiveness, and equitable distribution and use.
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Protocols, data dictionaries, and pre-determined messages about how MCMs are tested and approved, how EUAs work, etc. Systems Systems for effective communication with various audiences, including community stakeholders, public health partners, community health partners, health care providers, public, federal partners, university researchers, and other local and national partners.
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SOURCE: Adapted from the presentation "Federal Capabilities Needed to Monitor/Assess MCMs Used in Public Health Emergencies – An Initial Framework" made available by CDC, with additional examples offered by authors of this rapid expert consultation (in italic)
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. SOURCE: Adapted from the presentation "Federal Capabilities Needed to Monitor/Assess MCMs Used in Public Health Emergencies – An Initial Framework" made available by CDC, with additional examples offered by authors of this rapid expert consultation (in italic)
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Declaring states of "emergency," "disaster," or "PHE" – These declarations allow extensive federal powers to be invoked related to emerging infectious diseases and other substantial threats. By providing substantial liability protections and extensive preemption of conflicting state-based laws, real-time development and manufacturing of qualified MCMs can be facilitated through public–private partnerships.
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The ideas generated in this rapid expert consultation should be refined through a more careful and thoughtful analysis. The strengthening and pre-positioning of existing federal capabilities and the establishment of new federal capabilities to ensure robust data collection, analysis, translation, and communication to key stakeholders in the health care system are critical for the response to the ongoing COVID-19 pandemic and to future large-scale PHEs.
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2022b. Federal capabilities needed to monitor/assess MCMs used in public health emergencies -- an initial framework.
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https://www.ukri.org/news-and events/tackling-the-impact-of-covid-19/vaccines-and-treatments/recovery-trial-identifies covid-19-treatments (accessed April 12, 2022)
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government evaluation o Processes for receiving diagnostics at U.S. government labs for comparative evaluation o Agreements to consult with federal authorities regarding manufacturing reduction decisions Pre-developed specificity panels for diagnostics Pre-positioned protocols , pre-identified clinical trial networks, and related infrastructure (including information technology systems, experts, encryption protocols)
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to provide comparative and ongoing comparative evaluation of therapeutics/diagnostic real-world performance Ability to rapidly develop reference panels and specificity panels and make these widely available to support ongoing validation of diagnostic real-world performance Ability to collect and make available pooled patient samples (e.g., as raw materials for diagnostic panels and validation; challenge materials for ongoing MCM evaluation) Questions Considered for Each Pharmaceutical Medical Countermeasure What organization(s)
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How were the existing clinical networks (e.g., clinical trial networks, study and research networks) leveraged for the monitoring and assessment of this MCM?
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We extend gratitude to the staff of the National Academies, in particular Lisa Brown, Shalini Singaravelu, Kelsey Babik, Emma Fine, Matthew Masiello, and Margaret McCarthy, who contributed research, editing, and writing assistance. We are grateful to the Report Review Committee of the National Academies and to the following individuals for their review of this rapid expert consultation: Phyllis Arthur, Biotechnology Innovation Organization; Luciana Borio, ARCH Venture Partners, L.P.; Adrian F
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